An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Sponsor: Sarepta Therapeutics, Inc.
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
Time frame: Baseline, Month 12
Number of Participants Experiencing Acute Liver Injury (ALI)
Time frame: Baseline through Month 12
Secondary Endpoints
Time to Rise From Floor (Supine to Stand)
Loss of Ambulation (LOA)
Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score
Eligibility Criteria
Inclusion Criteria: * Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c): * Is at least 4 years of age at the time of infusion * Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval For Standard of Care Comparators (Cohort 2): * Is at least 4 years of age at the time of enrollment * Is unexposed to DMD gene therapy at the time of this observational study enrollment Exclusion Criteria: * Has any deletion of…
Read full criteria on CT.gov →✦ Analyst Commentary
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